Web Desc
Nucleic Acid Therapeutics: From Base Pairs to Bedsides
Organizer(s): Alan M. Gewirtz (in memoriam), Anastasia Khvorova and Mark A. Kay
Date: January 10 - 15, 2012
Location: Eldorado Hotel & Spa, Santa Fe, NM, USA
Sponsored by Isis Pharmaceuticals, Inc. and Regulus Therapeutics Inc.
For important information on the coronavirus, please click here
Summary of Meeting:
Small molecules and antibodies have revolutionized the treatment of cancer and appear promising for the treatment of many other diseases as well. Nonetheless, there are many candidate therapeutic targets that are not amenable to attack by the current generation of targeted therapies and, in a small but growing number of patients, resistance to initially successful targeted treatments evolves. This symposium will focus on the medicinal promise of pre- and post- transcriptional gene silencing (PPTGS) using oligodeoxynucleotides, small interfering RNA, microRNA and other nucleic acid based molecules. The meeting is motivated by the hypothesis that these molecules can be developed into effective drugs for the treatment of many common diseases that are collectively responsible for a great deal of human suffering. As this symposium will highlight, there is still much to do, but many in the field now hope that the time has finally arrived when “antisense” therapies will finally come of age and fulfill their promise as the magic bullets of the 21st century.
Scholarship Deadline: September 15 2011
Discounted Abstract Deadline: September 15 2011
Abstract Deadline: October 13 2011
Discounted Registration Deadline: November 10 2011
Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
Isis Pharmaceuticals, Inc.Regulus Therapeutics Inc.
We gratefully acknowledge additional support for this conference from:
Integrated DNA TechnologiesSigma Life Science
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

Isis Pharmaceuticals, Inc.

Prosensa Therapeutics

Regulus Therapeutics Inc.

RXi Pharmaceuticals

Tekmira


Xavier J. de Mollerat du Jeu
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations
Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations

Program

Tuesday, January 10 | 3:00PM - 7:30PM
Registration
Room: Zia Concourse


Tuesday, January 10 | 6:15PM - 7:15PM
Refreshments
Room: Zia Concourse


Tuesday, January 10 | 7:15PM - 8:00PM
Welcome and Introductory Remarks
Room: Anasazi Ballroom


Wednesday, January 11 | 7:00AM - 8:00AM
Breakfast
Room: Eldorado Court


Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 1 of 6
* Jennifer A. Doudna, HHMI/University of California, Berkeley, USA

Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 2 of 6
Bruce A. Sullenger, Duke University, USA
Controlling Thrombosis with Aptamers and Inflammation with Their Antidotes

Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 3 of 6
Robert J. Crouch, NICHD, National Institutes of Health, USA
Ribonucleases H: Function in Hydrolysis of RNA/DNA Hybrids

Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 4 of 6
John L. Rinn, University of Colorado Boulder, USA
Large Intergenic Non-Coding RNAs: From Discovery to Mechanism

Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 5 of 6
Jin Billy Li, Stanford University, USA
Generating an Atlas of Mammalian A-to-I RNA Editing

Wednesday, January 11 | 8:00AM - 11:00AM
Basic Mechanisms Underlining Oligonucleotide Therapeutic Dev
elopment

Room: Anasazi Ballroom

Speaker 6 of 6
Dongbo Yu, University of Texas Southwestern Medical Center, USA
Short Talk: Allele-Selective Inhibition of Huntingtin Expression by Single-Stranded RNAs that Act through the RNAi Pathway

Wednesday, January 11 | 9:20AM - 9:40AM
Coffee Break
Room: Zia Concourse


Wednesday, January 11 | 10:00AM - 10:00AM
On Own for Lunch and Recreation


Wednesday, January 11 | 11:15AM - 1:00PM
Poster Setup
Room: Sunset Room/Eldorado Court


Wednesday, January 11 | 1:00PM - 10:00PM
Poster Viewing
Room: Sunset Room/Eldorado Court


Wednesday, January 11 | 4:30PM - 5:00PM
Coffee Available
Room: Zia Concourse


Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 1 of 6
* Bruce A. Sullenger, Duke University, USA

Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 2 of 6
Anastasia Khvorova, University of Massachusetts Medical School, USA
Asymmetric, Hydrophobically Modified RNAi Compounds: From Mechanism of Action to Clinical Development

Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 3 of 6
Ryszard Kole, Ercole Biotech Consulting, USA
Eteplirsen, an Exon-Skipping Drug Candidate for Treatment of DMD

Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 4 of 6
Rasmus O. Bak, University of Aarhus, Denmark
Short Talk: Variable Potency and Transfer Efficiency of Lentivirus-Encoded miRNA Inhibitors

Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 5 of 6
Darren Wong, Medivation, Inc., USA
Short Talk: Antitumor Efficacy with Dicer Substrate siRNAs in the Absence of Immunostimulatory Effects

Wednesday, January 11 | 5:00PM - 7:00PM
Preclinical Developments
Room: Anasazi Ballroom

Speaker 6 of 6
Anita G. Seto, Expansion Therapeutics, USA
Short Talk: Discovery and Development of Novel Cardiovascular Drug Candidates Based on microRNA Targeting

Wednesday, January 11 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Eldorado Court/Sunset Room


Wednesday, January 11 | 7:30PM - 10:00PM
Poster Session 1
Room: Sunset Room/Eldorado Court


Thursday, January 12 | 7:00AM - 8:00AM
Breakfast
Room: Eldorado Court


Thursday, January 12 | 7:30AM - 8:00AM
Poster Setup
Room: Sunset Room/Eldorado Court


Thursday, January 12 | 8:00AM - 5:00PM
Poster Viewing
Room: Sunset Room/Eldorado Court


Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 1 of 6
* Larry Gold, SomaLogic Inc., USA

Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 2 of 6
Giles Campion, Prosensa Therapeutics, Netherlands
Systemic Antisense Treatment for Duchenne Muscular Dystrophy

Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 3 of 6
Richard S. Geary, Ionis Pharmaceuticals, Inc., USA
Role of Mipomersen in Potential Treatment of Severe Familial Hypercholesterolemia

Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 4 of 6
Ian MacLachlan, Tekmira Pharmaceuticals Corp., Canada
Integrating Preclinical and Clinical Experience in the Development of siRNA Products

Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 5 of 6
Eli Gilboa, University of Miami, Miller School of Medicine, USA
Aptamer-Targeted Modulation of Tumor Immunity

Thursday, January 12 | 8:00AM - 11:15AM
Clinical Applications I
Room: Anasazi Ballroom

Speaker 6 of 6
Elena Feinstein, Quark Pharmaceuticals Inc., Israel
Short Talk: siRNA Therapeutics for Eye Diseases

Thursday, January 12 | 9:20AM - 9:40AM
Coffee Break
Room: Zia Concourse


Thursday, January 12 | 11:15AM - 12:00PM
Lunch
Room: Eldorado Court/Sunset Room


Thursday, January 12 | 12:00PM - 2:30PM
Poster Session 2
Room: Sunset Room/Eldorado Court


Thursday, January 12 | 4:30PM - 5:00PM
Coffee Available
Room: Zia Concourse


Thursday, January 12 | 5:00PM - 5:50PM
Keynote Address
Room: Anasazi Ballroom

Speaker 1 of 2
* Mark A. Kay, Stanford University, USA

Thursday, January 12 | 5:00PM - 5:50PM
Keynote Address
Room: Anasazi Ballroom

Speaker 2 of 2
Craig C. Mello, University of Massachusetts Medical School, USA
RNA World and Therapeutic Applications

Thursday, January 12 | 5:50PM - 7:00PM
Structural Insights: Form Follows Function
Room: Anasazi Ballroom

Speaker 1 of 2
Jennifer A. Doudna, HHMI/University of California, Berkeley, USA
Dicing and Beyond: Regulatory RNA in Humans and Bacteria

Thursday, January 12 | 5:50PM - 7:00PM
Structural Insights: Form Follows Function
Room: Anasazi Ballroom

Speaker 2 of 2
Remo Rohs, University of Southern California, USA
DNA Shape is a Source of Protein-DNA Binding Specificity

Thursday, January 12 | 7:00PM - 7:00PM
On Own for Dinner


Friday, January 13 | 7:00AM - 8:00AM
Breakfast
Room: Eldorado Court


Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 1 of 6
* Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, USA

Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 2 of 6
Peter Sarnow, Stanford University School of Medicine, USA
Masking of the Hepatitis C Viral Genome by miR-122

Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 3 of 6
Jonathan S. Weissman, University of California, San Francisco, USA
Monitoring Protein Synthesis One Codon at a Time through Ribosome Profiling

Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 4 of 6
Stephen B. Baylin, Johns Hopkins University School of Medicine, USA
Methylation Mapping

Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 5 of 6
Larry Gold, SomaLogic Inc., USA
SOMAmers for Diagnostics and Therapeutics

Friday, January 13 | 8:00AM - 11:00AM
Novel Technologies in Oligonucleotide Target Discovery
Room: Anasazi Ballroom

Speaker 6 of 6
Shuo Gu, Stanford University, USA
Short Talk: Slicing Independent RISC Activation Requires the Argonaute Paz Domain

Friday, January 13 | 9:20AM - 9:40AM
Coffee Break
Room: Zia Concourse


Friday, January 13 | 10:00AM - 10:00AM
On Own for Lunch and Recreation


Friday, January 13 | 4:30PM - 5:00PM
Coffee Available
Room: Zia Concourse


Friday, January 13 | 5:00PM - 7:00PM
Clinical Applications II
Room: Anasazi Ballroom

Speaker 1 of 4
* Peter Sarnow, Stanford University School of Medicine, USA

Friday, January 13 | 5:00PM - 7:00PM
Clinical Applications II
Room: Anasazi Ballroom

Speaker 2 of 4
Dinah Sah, Voyager Therapeutics, USA
RNAi Therapeutics in Liver and Neurologic Diseases

Friday, January 13 | 5:00PM - 7:00PM
Clinical Applications II
Room: Anasazi Ballroom

Speaker 3 of 4
Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, USA
MicroRNA Therapeutics in Cancer

Friday, January 13 | 5:00PM - 7:00PM
Clinical Applications II
Room: Anasazi Ballroom

Speaker 4 of 4
Arthur A. Levin, Avidity Biosciences, USA
Targeting miR-122 for Treatment of Chronic Hepatitis C Infection with Miravirisen a 15- mer LNA-Modified AntimiR Oligonucleotide: From Bench to Bedside

Friday, January 13 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Eldorado Court/Sunset Room


Saturday, January 14 | 7:00AM - 8:00AM
Breakfast
Room: Eldorado Court


Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 1 of 7
* Arthur M. Krieg, Checkmate Pharmaceuticals, USA

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 2 of 7
Mark A. Kay, Stanford University, USA
RISC Loading of Duplex RNAs: Unwinding the Prospects for Antiviral Therapeutics

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 3 of 7
Christine Esau, Arcturus Therapeutics, USA
Targeting miR-33 for Atherosclerosis

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 4 of 7
Muthiah Manoharan, Alnylam Pharmaceuticals, Inc., USA
“The End of the Beginning:” Evolution of siRNA Conjugates as the Next Generation Systemic Delivery Approach for RNAi Therapeutics

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 5 of 7
Joshua T. Mendell, University of Texas Southwestern Medical Center, USA
MicroRNAs in Cancer: Biology and Therapeutic Applications

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 6 of 7
Ruth Broering, University Hospital of Essen, Germany
Short Talk: In vivo Application of LNP01-Formulated siRNAs Activates the Hepatic Immune System which can be Controlled by Chemical Ribose Modifications

Saturday, January 14 | 8:00AM - 11:15AM
Endogenous Small RNAs vs Exogenous Oligos
Room: Anasazi Ballroom

Speaker 7 of 7
Christopher J. Cheng, Yale University, USA
Short Talk: Cell-Penetrating Nanoparticles for Tumor-Targeted OncomiR Inhibition

Saturday, January 14 | 9:00AM - 9:20AM
Coffee Break
Room: Zia Concourse


Saturday, January 14 | 10:00AM - 10:00AM
On Own for Lunch and Recreation


Saturday, January 14 | 4:30PM - 5:00PM
Coffee Available
Room: Zia Concourse


Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 1 of 6
* Ian MacLachlan, Tekmira Pharmaceuticals Corp., Canada

Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 2 of 6
Brett P. Monia, Ionis Pharmaceuticals, Inc., USA
Broadening RNaseH-Based Antisense Pharmacology through Medicinal Chemistry

Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 3 of 6
Allen W. Nicholson, Temple University, USA
Alternative Mechanisms of Antisense Silencing by 2’-Fluoroarabinose Oligonucleotides

Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 4 of 6
Arthur M. Krieg, Checkmate Pharmaceuticals, USA

Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 5 of 6
Thazha P. Prakash, Ionis Pharmaceuticals, Inc., USA
Short Talk: Chemical Approaches for Achieving Activation of RNA Interference with Single Stranded Oligonucleotides in Animals

Saturday, January 14 | 5:00PM - 7:15PM
Novel Chemistries, Enhancing Enzymology and Animal Models in
Oligonucleotides Therapeutics Development

Room: Anasazi Ballroom

Speaker 6 of 6
Marcin T. Kortylewski, Beckman Research Institute, City of Hope, USA
Short Talk: Two-Pronged Approach to Human Cancer Immunotherapy using CpG-STAT3 siRNA Conjugates

Saturday, January 14 | 7:15PM - 8:15PM
Social Hour with Lite Bites
Room: Eldorado Court/Sunset Room


Saturday, January 14 | 8:00PM - 11:00PM
Entertainment
Room: Eldorado Court/Sunset Room


Saturday, January 14 | 8:00PM - 11:00PM
Cash Bar
Room: Eldorado Court/Sunset Room


Sunday, January 15 | 10:23AM - 10:23AM
Departure


*Session Chair
†Invited, not yet responded.